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Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A

NCT05265767 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2024-07-12

No results posted yet for this study

Summary

Factor VIII (FVIII) is a large plasma glycoprotein that participates in blood coagulation. Loss of circulating FVIII activity due to mutations within the F8 gene results in the X-linked, recessive bleeding disorder hemophilia A. The clinical presentation ranges from a mild to severe bleeding phenotype that correlates with the patient's residual plasma FVIII activity level.

Current state of the art treatment entails frequent infusion of FVIII protein. However, several limitations remain to treating hemophilia A, which are 1) access to FVIII-replacement products (currently \<30% of the world population is treated adequately, access is highly restricted in India), 2) high burden of compliance with treatment protocols particularly in children 3) the expense of FVIII-replacement products, 4) the development of humoral anti-FVIII immune responses that block FVIII activity and limit treatment efficacy and 5) morbidity due to crippling musculoskeletal disease when inadequately treated. Several newer hemostasis agents are being developed but like the recombinant Clotting Factor Concentrate (CFC) from the 1990s, these are also not likely to be made available in India for many years. Currently, the only cure for hemophilia A is orthotopic liver transplantation.

Conditions

Interventions

BIOLOGICAL

Auto CD34+PBSC transduced with a lentiviral vector encoding a novel coagulation factor VIII transgene

Auto CD34+PBSC transduced with a lentiviral vector encoding a novel coagulation factor VIII transgene administered by IV infusion following conditioning regimen.

Sponsors & Collaborators

  • Dr. H. Trent Spencer, Professor, Emory University of Medicine, Atlanta Ga, 30322

    collaborator UNKNOWN

  • Christian Medical College, Vellore, India

    lead OTHER

Principal Investigators

  • Alok Srivastava, MD · Christian Medical College, Vellore, India

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
45 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-04-01
Primary Completion
2024-06-28
Completion
2024-06-28
FDA Drug
Yes

Countries

  • India

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05265767 on ClinicalTrials.gov